Educational Resources in Pharmacoepidemiology (literature and videos)

General

Updated core competencies in pharmacoepidemiology to inform contemporary curricula and training for academia, government, and industry. Osborne V, Goodin A, Brown J, Winterstein AG, Bate A, Cohet C, Pont L, Moeny D, Klungel O, Pinheiro S, Seeger J, Chan KA, Edlavitch S, Tilson H, Layton D. Pharmacoepidemiol Drug Saf. 2024 Apr;33(4):e5789.

Where to begin? Thirty must-read papers for newcomers to pharmacoepidemiology. Pottegård A, Morin L, Hallas J, Gerhard T, Winterstein AG, Perez-Gutthann S, Tadrous M. Pharmacoepidemiol Drug Saf. 2022 Feb;31(2):257-259. 

Drug utilization

Core concepts in pharmacoepidemiology: Measures of drug utilization based on individual-level drug dispensing data. Rasmussen L, Wettermark B, Steinke D, Pottegård A. Pharmacoepidemiol Drug Saf. 2022 Oct;31(10):1015-1026.

Drug Utilization Research: Methods and Applications. Elseviers M, Wettermark B, Benkó R et al (editors), 2nd edition, 2024, John Wiley & Sons Ltd, Print ISBN: 9781119911654, Online ISBN: 9781119911685

Target trial emulation

Target Trial Emulation to Improve Causal Inference from Observational Data: What, Why, and How? Fu EL. J Am Soc Nephrol. 2023 Aug 1;34(8):1305-1314.

Using Big Data to Emulate a Target Trial When a Randomized Trial Is Not Available. Hernán MA, Robins JM. Am J Epidemiol. 2016 Apr 15;183(8):758-64.

Specifying a target trial prevents immortal time bias and other self-inflicted injuries in observational analyses. Hernán MA, Sauer BC, Hernández-Díaz S, Platt R, Shrier I. J Clin Epidemiol. 2016 Nov;79:70-75.

Emulating a target trial in case-control designs: an application to statins and colorectal cancer. Dickerman BA, García-Albéniz X, Logan RW, Denaxas S, Hernán MA. Int J Epidemiol. 2020 Oct 1;49(5):1637-1646

New user active comparator design

Core Concepts in Pharmacoepidemiology: New-User Designs. Her QL, Rouette J, Young JC, Webster-Clark M, Tazare J. Pharmacoepidemiol Drug Saf. 2024 Dec;33(12):e70048.

Core concepts in pharmacoepidemiology: Confounding by indication and the role of active comparators. Sendor R, Stürmer T. Pharmacoepidemiol Drug Saf. 2022 Mar;31(3):261-269.

Prevalent new user design

Prevalent new-user cohort designs for comparative drug effect studies by time-conditional propensity scores. Suissa S, Moodie EE, Dell’Aniello S. Pharmacoepidemiol Drug Saf. 2017 Apr;26(4):459-468.

The Prevalent New-user Design for Studies With no Active Comparator: The Example of Statins and Cancer. Suissa S, Dell’Aniello S, Renoux C. Epidemiology. 2023 Sep 1;34(5):681-689.

Clarifying the causal contrast: An empirical example applying the prevalent new user study design. Young JC, Webster-Clark M, Shmuel S, Garry EM, Mavros P, Stürmer T, Girman CJ. Pharmacoepidemiol Drug Saf. 2024 Apr;33(4):e5790. 

Initiator Types and the Causal Question of the Prevalent New-User Design: A Simulation Study. Webster-Clark M, Ross RK, Lund JL.Am J Epidemiol. 2021 Jul 1;190(7):1341-1348. 

Effect of combination treatment with glucagon-like peptide-1 receptor agonists and sodium-glucose cotransporter-2 inhibitors on incidence of cardiovascular and serious renal events: population based cohort study. Simms-Williams N, Treves N, Yin H, Lu S, Yu O, Pradhan R, Renoux C, Suissa S, Azoulay L. BMJ. 2024 Apr 25;385:e078242.

Self-controlled designs

Core Concepts: Self-Controlled Designs in Pharmacoepidemiology. Bots SH, Brown J, Wong AYS, Martin I, Douglas I, Klungel OH, Schultze A. Pharmacoepidemiol Drug Saf. 2025 Jan;34(1):e70071.

Special populations: pregnancy, pediatric, elderly

Use of real-world evidence from healthcare utilization data to evaluate drug safety during pregnancy Huybrechts KF, Bateman BT, Hernández-Díaz S. Pharmacoepidemiol Drug Saf 2019 Jul;28(7):906-922.

Studying early pregnancy exposures and outcomes in routinely collected healthcare data: Conceiving of target trials. Wood ME. Paediatr Perinat Epidemiol. 2024 Aug;38(6):482-485. 

Prenatal Antidepressant Exposure and the Risk of Attention-deficit/Hyperactivity Disorder in Childhood: A Cohort Study With Triangulation. Esen BÖ, Ehrenstein V, Sørensen HT, Laugesen K, Pedersen L. Epidemiology. 2022 Jul 1;33(4):581-592.

Propensity scores

Propensity scores for confounder adjustment when assessing the effects of medical interventions using nonexperimental study designs. Stürmer T, Wyss R, Glynn RJ, Brookhart MA. J Intern Med. 2014 Jun;275(6):570-80.

Biases

A Structural Description of Biases That Generate Immortal Time. Hernán MA, Sterne JAC, Higgins JPT, Shrier I, Hernández-Díaz S. Epidemiology. 2025 Jan 1;36(1):107-114.

Core concepts in pharmacoepidemiology: Key biases arising in pharmacoepidemiologic studies. Acton EK, Willis AW, Hennessy S. Pharmacoepidemiol Drug Saf. 2023 Jan;32(1):9-18.

Core Concepts in Pharmacoepidemiology: Quantitative Bias Analysis. Brown JP, Hunnicutt JN, Ali MS, Bhaskaran K, Cole A, Langan SM, Nitsch D, Rentsch CT, Galwey NW, Wing K, Douglas IJ. Pharmacoepidemiol Drug Saf. 2024 Oct;33(10):e70026.

Nordic registers

Nordic Health Registry-Based Research: A Review of Health Care Systems and Key Registries. Laugesen K, Ludvigsson JF, Schmidt M, Gissler M, Valdimarsdottir UA, Lunde A, Sørensen HT. Clin Epidemiol. 2021 Jul 19;13:533-554.

The Nordic countries as a cohort for pharmacoepidemiological research. Furu K, Wettermark B, Andersen M, Martikainen JE, Almarsdottir AB, Sørensen HT.Basic Clin Pharmacol Toxicol. 2010 Feb;106(2):86-94. 

Other papers in the Core Concepts in Pharmacoepidemiology – series of review articles in Pharmacoepidemiology and Drug Safety

Core Concepts in Pharmacoepidemiology: Principled Use of Artificial Intelligence and Machine Learning in Pharmacoepidemiology and Healthcare Research. Rough K, Rashidi ES, Tai CG, Lucia RM, Mack CD, Largent JA. Pharmacoepidemiol Drug Saf. 2024 Nov;33(11):e70041.

Core Concepts in Pharmacoepidemiology: Time-To-Event Analysis Approaches in Pharmacoepidemiology. Rippin G, Salmasi S, Sanz H, Largent J. Pharmacoepidemiol Drug Saf. 2024 Nov;33(11):e5886.

Core concepts in pharmacoepidemiology: Measurement of medication exposure in routinely collected healthcare data for causal inference studies in pharmacoepidemiology. Thai TN, Winterstein AG. Pharmacoepidemiol Drug Saf. 2024 Mar;33(3):e5683.

Core concepts in pharmacoepidemiology: Validation of health outcomes of interest within real-world healthcare databases. Weinstein EJ, Ritchey ME, Lo Re V 3rd. Pharmacoepidemiol Drug Saf. 2023 Jan;32(1):1-8.

Core concepts in pharmacoepidemiology: Fundamentals of the cohort and case-control study designs. Pottegård A. Pharmacoepidemiol Drug Saf. 2022 Aug;31(8):817-826.

Core concepts in pharmacoepidemiology: Violations of the positivity assumption in the causal analysis of observational data: Consequences and statistical approaches. Zhu Y, Hubbard RA, Chubak J, Roy J, Mitra N. Pharmacoepidemiol Drug Saf. 2021 Nov;30(11):1471-1485.

Miscellaneous

Encepp Guide.

Encepp Guide of Methodological Standards in Pharmacoepidemiology

Benchmarking and emulation

Prospective benchmarking of an observational analysis in the SWEDEHEART registry against the REDUCE-AMI randomized trial. Matthews AA, Dahebreh IJ, MacDonald CJ, Lindahl B, Hofmann R, Erlinge D, Yndigegn T, Berglund A, Jernberg T, Hernán MA. Eur J Epidemiol. 2024 Apr;39(4):349-361.

Emulation of Randomized Clinical Trials With Nonrandomized Database Analyses: Results of 32 Clinical Trials. Wang SV, Schneeweiss S; RCT-DUPLICATE Initiative; Franklin JM, Desai RJ, Feldman W, Garry EM, Glynn RJ, Lin KJ, Paik J, Patorno E, Suissa S, D’Andrea E, Jawaid D, Lee H, Pawar A, Sreedhara SK, Tesfaye H, Bessette LG, Zabotka L, Lee SB, Gautam N, York C, Zakoul H, Concato J, Martin D, Paraoan D, Quinto K. JAMA. 2023 Apr 25;329(16):1376-1385.

Reporting guidelines

Development of the TrAnsparent ReportinG of observational studies Emulating a Target trial (TARGET) guideline. Hansford HJ, Cashin AG, Jones MD, Swanson SA, Islam N, Dahabreh IJ, Dickerman BA, Egger M, Garcia-Albeniz X, Golub RM, Lodi S, Moreno-Betancur M, Pearson SA, Schneeweiss S, Sterne J, Sharp MK, Stuart EA, Hernan MA, Lee H, McAuley JH. BMJ Open. 2023 Sep 12;13(9):e074626.

The reporting of studies conducted using observational routinely collected health data statement for pharmacoepidemiology (RECORD-PE). Langan SM, Schmidt SA, Wing K, Ehrenstein V, Nicholls SG, Filion KB, Klungel O, Petersen I, Sorensen HT, Dixon WG, Guttmann A, Harron K, Hemkens LG, Moher D, Schneeweiss S, Smeeth L, Sturkenboom M, von Elm E, Wang SV, Benchimol EI. BMJ. 2018 Nov 14;363:k3532.

Peer review

A Road Map for Peer Review of Real-World Evidence Studies on Safety and Effectiveness of Treatments. Winterstein AG, Ehrenstein V, Brown JS, Stürmer T, Smith MY. Diabetes Care. 2023 Aug 1;46(8):1448-1454.

VIDEOS

Online introductory course: Introduction to pharmacoepidemiology. 10-part Youtube course by Anton Pottegård: https://www.youtube.com/playlist?list=PLLtSRj5IsMgTIKv8mSUy2r_9MISosnHVN

Nordic drug statistics

National drug statistics databases

Denmark: Sundhedsdatastyrelsen

Finland: Kela (only in Finnish) – Reports

Iceland: Directorate of Health (only in Icelandic)

Norway: Folkehelseinstituttet (only in Norwegian)

Sweden: Socialstyrelsen

Nordic (NOMESCO) drug statistics

In the Nordic Health and Welfare database, you can find detailed statistical data on the prevalence and sales of pharmaceutical products from the five Nordic countries (Denmark, Finland, Iceland, Norway, and Sweden) and the three autonomous areas (Faroe Islands, Greenland, and Åland). The statistics can be found under heading health.

The data are collected by NOMESCO. It is the Nordic Medico-Statistical Committee, a body under the Nordic Council of Ministers that coordinates and collects health statistics from the Nordic countries. Its primary goal is to create comparable health and welfare statistics across the region for policy-making and research.